Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce ABCA4 splice variants in Stargardt disease
Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Ida Maria Westin, Irina Golovleva, Guei‐Sheung Liu, Satheesh Kumar, Samuel McLenachan, Fred Kuanfu Chen, Fei‐Ting Hsu, Tom Edwards, Keith R G Martin, Albert W. Cheng, Raymond C.B. Wong
This study evaluates RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce mis-spliced ABCA4 variants in Stargardt disease, showing significant reductions in disease-causing splice variants.
bioRxiv · Mar 8, 2024 · PreprintMolecular Biology